What it is & clinical trials
What is Alström Syndrome?
Alström syndrome is a rare, inherited, autosomal recessive syndrome resulting from the mutation of the ALMS1 gene that leads to childhood or adolescent obesity, type 2 diabetes with severe insulin resistance, dyslipidemia, hypertension and severe multi-organ fibrosis, involving the heart, liver and kidney.
The most common cause of death in patients with Alström syndrome is heart failure with dilated cardiomyopathy due to progressive cardiac fibrosis. Fibrosis leading to liver failure is also responsible for a large number of deaths. Alström syndrome is also characterized by a progressive loss of vision and hearing and short stature.
Alström syndrome affects over 800 patients worldwide. There are no therapies currently approved to treat Alström syndrome.
Development stage & clinical trial information:
The Phase 2, open-label safety and tolerability study of fezagepras in subjects with Alström Syndrome (ALMS) [NCT02739217], was completed in June 2018 and indicated that fezagepras (PBI-4050) was safe and well tolerated by the ALMS subjects in the trial when treated with 800 mg of fezagepras once daily. An open-label rollover, single-arm, Phase 2 clinical trial of fezagepras for the ongoing treatment of ALMS patients was terminated in May 2020, as a result of the constraints of the COVID-19 pandemic. Nine ALMS patients completed more than two years of treatment with fezagepras.
The trial was performed at the specialty center for the care of UK patients with Alström syndrome at the Queen Elizabeth Hospital, Birmingham, UK.
Fezagepras has been granted Orphan Drug Designation by the FDA and the EMA for the treatment of Alström syndrome as well as being granted the Promising Innovative Medicine (PIM) designation by the UK Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of Alström syndrome.